Regenxbio Meets Primary Endpoint in Duchenne Muscular Dystrophy Study

Dow Jones

05/14

By Nicholas G. Miller

Regenxbio said top-line results showed it met its primary endpoint in a Phase 3 study of its RGX-202 gene therapy for Duchenne Muscular Dystrophy.

The trial met its primary endpoint with high statistical significance, with 93% of participants reaching at least 10% microdystrophin expression, which refers to the production of a particular version of a protein to treat DMD, at Week 12. RGX-202 also demonstrated a statistically significant correlation between microdystrophin expression and interim functional improvement.

"Today's top-line results underscore how our novel construct and differentiated therapeutic approach support a favorable safety profile and potential clinical benefit, including in older patients where progressive decline is expected," said Chief Medical Officer Steve Pakola.

The company plans to pursue accelerated approval for RGX-202 and is preparing for a potential commercial launch in 2027.

Write to Nicholas G. Miller at nicholas.miller@wsj.com.

(END) Dow Jones Newswires

May 14, 2026 08:06 ET (12:06 GMT)

Copyright (c) 2026 Dow Jones & Company, Inc.

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Miller \n</p>\n<pre style=\"white-space: pre\">\n \n</pre>\n<p>\n  Regenxbio said top-line results showed it met its primary endpoint in a Phase 3 study of its RGX-202 gene therapy for Duchenne Muscular Dystrophy. \n</p>\n<p>\n  The trial met its primary endpoint with high statistical significance, with 93% of participants reaching at least 10% microdystrophin expression, which refers to the production of a particular version of a protein to treat DMD, at Week 12. RGX-202 also demonstrated a statistically significant correlation between microdystrophin expression and interim functional improvement. \n</p>\n<p>\n  \"Today's top-line results underscore how our novel construct and differentiated therapeutic approach support a favorable safety profile and potential clinical benefit, including in older patients where progressive decline is expected,\" said Chief Medical Officer Steve Pakola. \n</p>\n<p>\n  The company plans to pursue accelerated approval for RGX-202 and is preparing for a potential commercial launch in 2027. \n</p>\n<pre style=\"white-space: pre\">\n \n</pre>\n<p>\n  Write to Nicholas G. Miller at nicholas.miller@wsj.com. \n</p>\n<pre style=\"white-space: pre\">\n \n</pre>\n<p>\n  (END) Dow Jones Newswires\n</p>\n<p>\n  May 14, 2026 08:06 ET (12:06 GMT)\n</p>\n<p>\n  Copyright (c) 2026 Dow Jones &amp; Company, Inc.\n</p>\n</font>\n\n</article>\n</div>\n</body>\n</html>\n","isBrief":false,"type":0,"news_type":1,"symbol":"RGNX","symbol_name":"Regenxbio Inc","start_time":0,"source_url":"https://dowjonesnews.com/newdjn/logon.aspx?AL=N","article_id":"2635712995","we_media_id":"106","thumbnails":[],"rights":null,"url":"https://stock-news.laohu8.com/highlight/detail?id=2635712995","pubTimestamp":1778760360,"columns":[],"sourceInfo":null,"weMediaInfo":{"media_name":"Dow Jones","introduction":"Dow Jones publishes the world’s most trusted business news and financial information in a variety of media.","home_visible":0,"id":"106","head_image":"https://static.tigerbbs.com/150f88aa4d182df19190059f4a365e99"},"summary":"Regenxbio said top-line results showed it met its primary endpoint in a Phase 3 study of its RGX-202 gene therapy for Duchenne Muscular Dystrophy.The trial met its primary endpoint with high statistical significance, with 93% of participants reaching at least 10% microdystrophin expression, which refers to the production of a particular version of a protein to treat DMD, at Week 12. 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Miller \n\n\n \n\n\n  Regenxbio said top-line results showed it met its primary endpoint in a Phase 3 study of its RGX-202 gene therapy for Duchenne Muscular Dystrophy. \n\n\n  The trial met its primary endpoint with high statistical significance, with 93% of participants reaching at least 10% microdystrophin expression, which refers to the production of a particular version of a protein to treat DMD, at Week 12. RGX-202 also demonstrated a statistically significant correlation between microdystrophin expression and interim functional improvement. \n\n\n  \"Today's top-line results underscore how our novel construct and differentiated therapeutic approach support a favorable safety profile and potential clinical benefit, including in older patients where progressive decline is expected,\" said Chief Medical Officer Steve Pakola. \n\n\n  The company plans to pursue accelerated approval for RGX-202 and is preparing for a potential commercial launch in 2027. \n\n\n \n\n\n  Write to Nicholas G. Miller at nicholas.miller@wsj.com. \n\n\n \n\n\n  (END) Dow Jones Newswires\n\n\n  May 14, 2026 08:06 ET (12:06 GMT)\n\n\n  Copyright (c) 2026 Dow Jones & Company, Inc.","kind":"highlight","is_publish_news":true,"is_publish_highlight":false,"is_publish_live":false,"is_publish_wemedia":true,"editions":null,"column":"","sentiment":"1","news_tag":"dataReport","news_rank":0,"isVideo":false,"video":null,"symbols":[],"gpt_button":0,"need_auth":true,"need_login_tip":false,"code":"91000000","status":"200"}}}